E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Subacute cutaneous lupus erythematosus, in patients who do not respond to first line therapies (i.e. antimalrials) |
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E.1.1.1 | Medical condition in easily understood language |
Autoimmune disease characterized by clear cut boarderd, red, round and/or scaly leasions on the body areas which are exposed to sun. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.0 |
E.1.2 | Level | HLT |
E.1.2 | Classification code | 10025135 |
E.1.2 | Term | Lupus erythematosus (incl subtypes) |
E.1.2 | System Organ Class | 10028395 - Musculoskeletal and connective tissue disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Assess preliminary clinical efficacy of oral KRP203 in treatment refractory patients with subacute cutaneous lupus erythematosus (SCLE) after 12 weeks of treatment with KPR203 versus placebo. |
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E.2.2 | Secondary objectives of the trial |
• Assess safety and tolerability of oral KRP203 in patients with SCLE
• Assess steady-state blood concentrations of KRP203 and KRP203-Phosphate (KRP203-P) in SCLE patients
• Assess changes in the activity of SCLE using visual analogue scales for global skin health as assessed by the physician and the patient
• Assess the systemic features of SCLE using the Systemic Lupus Erythematosus Disease Activity Index (SLEDAI)
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Male and female (non-pregnant, non-lactating female)patients between 18-65 years of age. Having SCLE as described by Sontheimer et al (see Section 6.2) for at least 3 months before screening.
- Moderate to severe active skin disease at baseline by having an activity score of CLASI ³ 6, has to be demontrated, with at least 2 points in at least 3 different anatomical locations for erythema or scale/hypertrophy.
Other protocol-defined inclusion criteria may apply
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E.4 | Principal exclusion criteria |
- Patients with preexisting nephritis, central nervous or pulmonary involvement or any major internal organ damage, either related or unrelated to lupus. Patients having signs or symptoms of other autoimmune diseases such as systemic lupus erythematosus or Sjogren`s syndrome are allowed to enter the study
- Pregnant, planning to get pregnant, and/or lactating females or males planning to father a child within time period of the study or subsequent exclusionary period.
Other protocol-defined exclusion criteria may apply.
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E.5 End points |
E.5.1 | Primary end point(s) |
Mean difference from baseline in the activity score of CLASI (from now on referred to only as CLASI). |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
For the purposes of assessing the sample size, efficacy would be demonstrated if there would be at least 70% confidence that the true improvement induced by KRP203 in mean CLASI at 12 weeks is at least 5 points and at least 70% confidence that KRP203 is superior to placebo in terms of the primary endpoint.
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E.5.2 | Secondary end point(s) |
- Safety and tolerability of KRP203A as assessed by standard safety evaluations and the use of SLEDAI
- Steady state PK blood concentrations of KRP203 and KRP203-p assessed by LC-MS/MS method wit a LLQ of 0.05 ng/mL
- Assess changes in the activity of SCLE using visual analogue scales for global skin health as assessed by the physician and the patient |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Yes |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 9 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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Last patient last visit (LPLV): January 2012 |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |